LB Pharmaceuticals (LBRX) 25th Annual Needham Virtual Healthcare Conference summary

Strategic vision and pipeline

• Aims to become a fully integrated CNS-focused company, ready to launch therapeutics upon approval.

• LB-102, a late-stage asset, targets schizophrenia, bipolar depression, and adjunctive MDD, with a streamlined path to approval in schizophrenia after FDA engagement.

• Robust IP portfolio extends to 2041/2042, with strong funding secured through Q2 2029, supporting all planned clinical milestones.

• Multiple clinical trials are fully funded: phase III in schizophrenia (data H2 2027), phase II in bipolar depression (data Q1 2028), and adjunctive MDD (data H1 2029).

• Opportunities exist for expansion into additional indications, including negative symptoms, Alzheimer's disease psychosis, and agitation.

Clinical development and trial design

• Phase III schizophrenia trial initiated, targeting 460 patients, with primary endpoint PANSS change at day 42 and cognition as a secondary endpoint.

• Open-label extension will enroll ~900 patients for 52 weeks to accrue safety data and analyze cognition and negative symptoms.

• Phase II bipolar depression trial is a monotherapy, fixed-flexible dose design, enrolling 320 patients, with MADRS-10 as the primary endpoint.

• Adjunctive MDD trial will enroll 380 patients across US/EU, using a similar design with dose escalation based on response.

• All trials include prospective measurement of cognition and anhedonia.

Differentiated mechanism and competitive positioning

• LB-102 is a selective D2, D3, and 5-HT7 inhibitor, with improved brain penetration and once-daily dosing due to methylation of amisulpride.

• Demonstrates rapid onset, sustained response, and potential class-leading safety, including low EPS and minimal prolactin/QT effects.

• Unique presynaptic autoreceptor engagement enables dose-dependent effects for psychosis and mood disorders.

• Competes favorably with muscarinic antipsychotics due to simpler dosing, no food effect, and fewer side effects.

• Real-world amisulpride data and clinical experience support dose selection and efficacy expectations for LB-102.